المؤلفون: Peyvandi, Flora, Cataland, Spero, Scully, Marie, Coppo, Paul, Knoebl, Paul, Kremer Hovinga, Johanna A., Metjian, Ara, de la Rubia, Javier, Pavenski, Katerina, Minkue Mi Edou, Jessica, De Winter, Hilde, Callewaert, Filip

المصدر: Peyvandi, Flora; Cataland, Spero; Scully, Marie; Coppo, Paul; Knoebl, Paul; Kremer Hovinga, Johanna A.; Metjian, Ara; de la Rubia, Javier; Pavenski, Katerina; Minkue Mi Edou, Jessica; De Winter, Hilde; Callewaert, Filip (2021). Caplacizumab prevents refractoriness and mortality in acquired thrombotic thrombocytopenic purpura: integrated analysis. Blood advances, 5(8), pp. 2137-2141. American Society of Hematology 10.1182/bloodadvances.2020001834

مصطلحات موضوعية: 610 Medicine & health

الوصف: The efficacy and safety of caplacizumab in individuals with acquired thrombotic thrombocytopenic purpura (aTTP) have been established in the phase 2 TITAN and phase 3 HERCULES trials. Integrated analysis of data from both trials was conducted to increase statistical power for assessing treatment differences in efficacy and safety outcomes. Caplacizumab was associated with a significant reduction in the number of deaths (0 vs 4; P < .05) and a significantly lower incidence of refractory TTP (0 vs 8; P < .05) vs placebo during the treatment period. Consistent with the individual trials, treatment with caplacizumab resulted in a faster time to platelet count response (hazard ratio, 1.65; P < .001), a 72.6% reduction in the proportion of patients with the composite end point of TTP-related death, TTP exacerbation, or occurrence of at least 1 treatment-emergent major thromboembolic event during the treatment period (13.0% vs 47.3%; P < .001), and a 33.3% reduction in the median number of therapeutic plasma exchange days (5.0 vs 7.5 days) vs placebo. No new safety signals were identified; mild mucocutaneous bleeding was the main safety finding. This integrated analysis provided new evidence that caplacizumab prevents mortality and refractory disease in acquired TTP and strengthened individual trial findings, with a confirmed favorable safety and tolerability profile. These trials were registered at www.clinicaltrials.gov as #NCT01151423 and #NCT02553317.

وصف الملف: application/pdf

العلاقة: https://boris.unibe.ch/156090Test/

الإتاحة: https://doi.org/10.1182/bloodadvances.2020001834Test
https://boris.unibe.ch/156090/1/Caplacizumab_prevents_advancesadv2020001834.pdfTest
https://boris.unibe.ch/156090Test/

المؤلفون: Koh, Youngil, Ghobrial, Irene, Martinez-Lopez, Joaquin, Rodriguez-Otero, Paula, Lepine, Lucie, Mace, Sandrine, Oprea, Corina, Mateos, Maria-Victoria, Devisme, Christine, Dubin, Franck, Schjesvold, Fredrik, Peceliunas, Valdas, Leleu, Xavier, Kim, Jin Seok, Besisik, Sevgi Kalayoglu, Hungria, Vania, Hermansen, Emil, de la Rubia, Javier, Quach, Hang, Prince, Miles, Parmar, Gurdeep

المساهمون: Harvard University ,, 2770412

مصطلحات موضوعية: Onkoloji, Sağlık Bilimleri, Tıp, HEMATOLOJİ, Klinik Tıp (MED), Klinik Tıp, ONKOLOJİ, Health Sciences, Hematology, Oncology, Hematoloji, İç Hastalıkları, Dahili Tıp Bilimleri

العلاقة: CLINICAL LYMPHOMA MYELOMA & LEUKEMIA; Ghobrial I., Rodriguez-Otero P., Koh Y., Martinez-Lopez J., Parmar G., Prince M., Quach H., de la Rubia J., Hermansen E., Hungria V., et al., "ITHACA, a randomized multicenter phase 3 study of Isatuximab in combination with Lenalidomide and Dexamethasone in high-risk smoldering Multiple Myeloma: safety run-in preliminary results", CLINICAL LYMPHOMA MYELOMA & LEUKEMIA, cilt.21, 2021; vv_1032021; av_be09d9a7-e1ea-476f-8197-884cf8344e0e; http://hdl.handle.net/20.500.12627/173933Test; 21

الإتاحة: https://doi.org/20.500.12627/173933Test
https://hdl.handle.net/20.500.12627/173933Test

المؤلفون: Pascual‐Izquierdo, Cristina, del Rio‐Garma, Julio, de la Rubia, Javier, Viejo, Aurora, Mingot, Eva, Cid, Joan, Solanich, Xavier, Fernández‐Sojo, Jesús, Martín‐Sánchez, Jesús, Hernández, Luis, García‐Gala, José María, Alonso, Nieves, González, Victoria, Oliva, Ana, Gómez‐Seguí, Inés, Goterris, Rosa, Guerra, Luisa, García‐Candel, Faustino, Fernández‐Docampo, Marta, Antelo, María Luisa, Salgado‐Barreira, Ángel, Salinas, Ramón

المصدر: Journal of Clinical Apheresis ; volume 36, issue 4, page 563-573 ; ISSN 0733-2459 1098-1101

الوصف: Background Immune‐mediated thrombotic thrombocytopenic purpura (iTTP) is a rare disease characterized by the presence of anti‐ADAMTS13 autoantibodies. Achieving accurate information on incidence and customary disease management is important to provide appropriate diagnostic and therapeutic resources. The aim of this study was to determine the incidence and outcomes of iTTP in Spain. Study design and methods A cross‐sectional survey was carried out among Spanish hospitals, focused on iTTP patients ≥16 years old attended between 2015 and 2017, and those at follow‐up before that interval. Incidence, prevalence, mortality, refractoriness, exacerbations, treatment complications, relapses, and sequelae were estimated. Results Forty‐two hospitals covering roughly 20 million inhabitants answered the survey and reported 203 episodes (138 newly‐diagnosed and 65 relapses), of which 193 (95.1%) were treated. Incidence was 2.67 (95% CI 1.90‐3.45) patients per million inhabitants per year and prevalence 21.44 (95% CI% 19.10‐23.73) patients per million inhabitants. At diagnosis, ADAMTS13 activity and anti‐ADAMTS13 autoantibody were measured in 97% and 84.3% of reported episodes, respectively. Fifteen patients (7.4%) died as a direct consequence of iTTP, 6 of them before receiving any iTTP‐specific treatment. Thirty‐one (16.1%) of the 193 treated episodes were refractory to plasma exchange and corticosteroids, and 51 (26.4%) suffered at least one exacerbation. Conclusion iTTP incidence and prevalence were somewhat higher than those documented in neighboring countries. Together with data on treatments and outcomes, this information will allow us to better estimate what is needed to improve diagnosis and prognosis of iTTP patients in Spain.

الإتاحة: https://doi.org/10.1002/jca.21894Test

المؤلفون: del Río-Garma, Julio, Bobillo, Sabela, de la Rubia, Javier, Pascual, Cristina, García-Candel, Faustino, García-Gala, Jose M., Gonzalez, Reyes, Abril, Laura, Vidan, Julia, Gomez, Maria Jesús, Peña, Francisco, Arbona, Cristina, Martín-Sanchez, Jesús, Moreno, Gemma, Romón, Iñigo, Viejo, Aurora, Oliva, Ana, Linares, Mónica, Salinas, Ramón, Pérez, Sonia, Garcia-Erce, Jose A., Pereira, Arturo

المصدر: Annals of Hematology ; volume 101, issue 1, page 59-67 ; ISSN 0939-5555 1432-0584

مصطلحات موضوعية: Hematology, General Medicine

الإتاحة: https://doi.org/10.1007/s00277-021-04685-8Test

المؤلفون: Cejalvo, María José, Bustamante, Gabriela, González, Esther, Vázquez-Álvarez, Judith, García, Ricarda, Ramírez-Payer, Ángel, Pérez-Persona, Ernesto, Abella, Eugenia, Garzón, Sebastián, García, Antoni, Jarque, Isidro, González, Marta Sonia, Sampol, Antonia, Motlló, Cristina, Martí, Josep María, Alcalá, Magdalena, Duro, Rafael, González, Yolanda, Sastre, José Luis, Sarrà, Josep, Lostaunau, Giselle, López, Rocío, de la Rubia, Javier

المساهمون: Celgene, a Bristol Myers Squibb Company

المصدر: Annals of Hematology ; volume 100, issue 7, page 1769-1778 ; ISSN 0939-5555 1432-0584

مصطلحات موضوعية: Hematology, General Medicine

الإتاحة: https://doi.org/10.1007/s00277-021-04529-5Test

المؤلفون: Ghobrial, Irene, Rodríguez-Otero, Paula, Koh, Youngil, Martínez-López, Joaquín, Parmar, Gurdeep, Prince, Miles, Quach, Hang, de la Rubia, Javier, Hermansen, Emil, Hungria, Vania, Besisik, Sevgi Kalayoglu, Kim, Jin Seok, Leleu, Xavier, Peceliunas, Valdas, Schjesvold, Fredrik, Dubin, Franck, Devisme, Christine, Lepine, Lucie, Macé, Sandrine, Oprea, Codina, Mateos, María-Victoria

المصدر: Clinical Lymphoma Myeloma and Leukemia ; volume 21, page S109-S110 ; ISSN 2152-2650

مصطلحات موضوعية: Cancer Research, Oncology, Hematology

الإتاحة: https://doi.org/10.1016/s2152-2650Test(21)02264-3
https://api.elsevier.com/content/article/PII:S2152265021022643?httpAccept=text/xmlTest
https://api.elsevier.com/content/article/PII:S2152265021022643?httpAccept=text/plainTest

المؤلفون: Ruiz, Raquel García, Martínez, Elena Meseguer, Castillo, María Jiménez, Verdet, María Consejo Ortí, Aracil, Eva Francés, Herraiz, Mario Arnao, Ortega, Omara Samantha Cortés, Alcaina, Pilar Solves, Andújar, María José Cejalvo, de la Piedra, Rafael Andreu, Feria, Ana García, García, María Paz Ribas, de la Rubia, Javier, Vicente, Irene Navarro

المصدر: Clinical Lymphoma Myeloma and Leukemia ; volume 21, page S126 ; ISSN 2152-2650

مصطلحات موضوعية: Cancer Research, Oncology, Hematology

الإتاحة: https://doi.org/10.1016/s2152-2650Test(21)02292-8
https://api.elsevier.com/content/article/PII:S2152265021022928?httpAccept=text/xmlTest
https://api.elsevier.com/content/article/PII:S2152265021022928?httpAccept=text/plainTest

المؤلفون: Moreno, David, Oriol, A, de la Rubia, Javier, Hernández, Miguel Teodoro, Iñigo, Belen, Palomera, Luis, de Arriba, Felipe, González, Yolanda, Teruel, Ana Isabel, Granell, Miquel, de la Guía, Ana López, Mayol, Antonia Sampol, Ríos, Rafael, Sureda, A, Gutierrez, Norma C., Calasanz, María José, Ramos, María Luisa Martin, Mateos, María-Victoria, San-Miguel, Jesús F., Lahuerta, Juan José, Bladé, J, Rosiñol, Laura

المصدر: Clinical Lymphoma Myeloma and Leukemia ; volume 21, page S134-S135 ; ISSN 2152-2650

مصطلحات موضوعية: Cancer Research, Oncology, Hematology

الإتاحة: https://doi.org/10.1016/s2152-2650Test(21)02305-3
https://api.elsevier.com/content/article/PII:S2152265021023053?httpAccept=text/xmlTest
https://api.elsevier.com/content/article/PII:S2152265021023053?httpAccept=text/plainTest

المؤلفون: Garcia-Guiñón, Antonio, Morales, Enrique, Sureda, A, González-García, Esther, Durán, María Soledad, Escalante, Fernando, Gironella, M, González-López, Tomás José, Duro, Rafael, Encinas, Manuel Pérez, Ribas, Paz, Cabezudo, Elena, García, Rafael Lluch, Español, Ignacio, Cabanas, Valentín, García-Muñoz, Ricardo, Márquez, José Antonio, Navarro, Mireya, de la Rubia, Javier

المصدر: Clinical Lymphoma Myeloma and Leukemia ; volume 21, page S97-S98 ; ISSN 2152-2650

مصطلحات موضوعية: Cancer Research, Oncology, Hematology

الإتاحة: https://doi.org/10.1016/s2152-2650Test(21)02242-4
https://api.elsevier.com/content/article/PII:S2152265021022424?httpAccept=text/xmlTest
https://api.elsevier.com/content/article/PII:S2152265021022424?httpAccept=text/plainTest

المؤلفون: Izquierdo, Cristina Pascual, Mingot-Castellano, María Eva, Fuentes, Ana E. Kerguelen, García-Arroba Peinado, José, Cid, Joan, Jimenez, Maria Moraima, Valcarcel, David, Gómez-Seguí, Inés, de la Rubia, Javier, Martin, Paz, Goterris, Rosa, Hernández, Luis, Tallón, Inmaculada, Varea, Sara, Fernández, Marta, García-Muñoz, Nadia, Vara, Míriam, Zarzoso, Miguel Fernández, García-Candel, Faustino, Paciello, María Liz, García-García, Irene, Zalba, Saioa, Campuzano, Verónica, Gala, José María, Estévez, Julia Vidán, Jiménez, Gemma Moreno, López Lorenzo, José Luis, Arias, Elena González, Freiría, Carmen, Solé, María, Ávila Idrovo, Laura Francisca, Hernández Castellet, José Carlos, Cruz, Naylen, Lavilla, Esperanza, Pérez-Montaña, Albert, Atucha, Jon Ander, Moreno Beltrán, María Esperanza, Moreno Macías, Juán Ramón, Salinas, Ramón, del Rio-Garma, Julio

المصدر: Blood Advances; December 2022, Vol. 6 Issue: 24 p6219-6227, 9p

مستخلص: Immune thrombotic thrombocytopenic purpura (iTTP) is a thrombotic microangiopathy caused by anti-ADAMTS13 antibodies. Caplacizumab is approved for adults with an acute episode of iTTP in conjunction with plasma exchange (PEX) and immunosuppression. The objective of this study was to analyze and compare the safety and efficacy of caplacizumab vs the standard of care and assess the effect of the concomitant use of rituximab. A retrospective study from the Spanish TTP Registry of patients treated with caplacizumab vs those who did not receive it was conducted. A total of 155 patients with iTTP (77 caplacizumab, 78 no caplacizumab) were included. Patients initially treated with caplacizumab had fewer exacerbations (4.5% vs 20.5%; P < .05) and less refractoriness (4.5% vs 14.1%; P < .05) than those who were not treated. Time to clinical response was shorter when caplacizumab was used as initial treatment vs caplacizumab used after refractoriness or exacerbation. The multivariate analysis showed that its use in the first 3 days after PEX was associated with a lower number of PEX (odds ratio, 7.5; CI, 2.3-12.7; P < .05) and days of hospitalization (odds ratio, 11.2; CI, 5.6-16.9; P < .001) compared with standard therapy. There was no difference in time to clinical remission in patients treated with caplacizumab compared with the use of rituximab. No severe adverse event was described in the caplacizumab group. In summary, caplacizumab reduced exacerbations and refractoriness compared with standard of care regimens. When administered within the first 3 days after PEX, it also provided a faster clinical response, reducing hospitalization time and the need for PEX.